2.7.6 Synopsis of Individual Studies : Appendix

- 138 -

CSL Behring 2.7.6 Synopsis of Individual Studies: Appendix Recombinant Fusion Protein Linking Coagulation Factor IX with Albumin (rIX-FP)

Table of Contents Table of Contents ....................................................................................................................... 2

A.1 Study 2001 ......................................................................................................................... 4

Table A.1-1 Study 2001: Summary of Treatment-Emergent Adverse Events by System Organ Class and Preferred Term (Safety Population) ........................................................ 4

Table A.1-2 Study 2001: Summary of Treatment-Emergent Adverse Events by System Organ Class Preferred Term, and Maximum Relationship to Study Treatment (Safety Population) ......................................................................................................................... 6

A.2 Study 2004 ....................................................................................................................... 16

Table A.2-1 Study 2004: Summary Statistics of Uncorrected FIX Activity in Plasma (IU/dL) (PK Population) .................................................................................................. 16

Table A.2-2 Study 2004: Annualized Bleeding Rate by Type of Bleed and Treatment Modality: Treatment Period (Efficacy Population) ......................................................... 19

Table A.2-3 Study 2004: Summary of Minor and Moderate Bleeding Episodes by Treatment Modality: Treatment Period (PP Population) ................................................. 35

Table A.2-4 Study 2004: Disease History by Treatment Modality (All Populations) ......... 42

Table A.2-5 Study 2004: Number of rIX-FP Infusions Required to Control Bleeding Episode by Severity: Treatment Period (Efficacy and PP Populations) .......................... 58

Table A.2-6 Study 2004: Investigator’s Clinical Assessment of Hemostatic Efficacy for Treatment of Minor Moderate Bleeding Episodes: Treatment Period (Efficacy and PP Population) ....................................................................................................................... 73

Table A.2-7 Study 2004: Consumption of rIX-FP During Routine Prophylaxis: Treatment Period (Efficacy and PP Populations) .............................................................................. 76

Table A.2-8 Study 2004: Dose Assignment and Dose Adjustment by Treatment Modality and Country ...................................................................................................................... 80

Table A.2-9 Study 2004: Consumption of rIX-FP During Surgical Prophylaxis: Treatment Period (Efficacy and PP Population) ................................................................................ 85

Table A.2-10 Study 2004: Derived Efficacy Variables by Subject..................................... 88

Table A.2-11 Study 2004: Consumption of rIX-FP for the Treatment of Bleeding Episodes: Treatment Period (Efficacy and PP Populations) ........................................... 91

Table A.2-12 Study 2004: Summary of Treatment-Emergent Adverse Events by System Organ Class and Preferred Term (Safety Population) ...................................................... 94

Table A.2-13 Study 2004: Summary of Treatment-Emergent Adverse Events by System Organ Class, Preferred Term, and Maximum Relationship to Study Treatment (Safety Population) ....................................................................................................................... 96

A.3 Study 3001 ..................................................................................................................... 105

Table A.3-1 Study 3001: Annualized Spontaneous Bleeding Rate (Primary Efficacy Population) ..................................................................................................................... 105

- 139 -

CSL Behring 2.7.6 Synopsis of Individual Studies: Appendix Recombinant Fusion Protein Linking Coagulation Factor IX with Albumin (rIX-FP)

Table A.3-2 Study 3001: Annualized Spontaneous Bleeding Rate Compared Between 7-Day and 10-Day Prophylaxis Regimens (Efficacy Population) ..................................... 108

Table A.3-3 Study 3001: Number of rIX-FP Infusions Required to Achieve Hemostasis by Cause (Efficacy Population) .......................................................................................... 117

Table A.3-4 Study 3001: Number of rIX-FP Infusions Required to Achieve Hemostasis by Location (Efficacy Population) ...................................................................................... 123

Table A.3-5 Study 3001: Hemoglobin Levels Relative to Surgery Time (Surgical Population) ..................................................................................................................... 125

Table A.3-6 Study 3001: Summary of Dose Assignment and Dose Adjustment (Safety Population) ..................................................................................................................... 130

Table A.3-7 Study 3001: Inhibitors and Antibodies (Safety Population) .......................... 134

Table A.3-8 Study 3001: Treatment-Emergent Adverse Events by System Organ Class and Preferred Term (Safety Population) ............................................................................... 135

Table A.3-9 Study 3001: Treatment-Emergent Adverse Events by System Organ Class, Preferred Term, and Maximum Relationship (Safety Population) ................................. 145

Table A.3-10 Study 3001: Japanese Treatment-Emergent Adverse Events by System Organ Class and Preferred Term (Japanese Safety Population) ..................................... 184

Table A.3-11 Study 3001: Japanese Related Treatment-Emergent Adverse Events by System Organ Class and Preferred Term (Japanese Safety Population) ........................ 188

A.4 Study 3002 ..................................................................................................................... 189

Table A.4-1 Study 3002: Summary of Bleeding Episodes Requiring Treatment by Bleeding Type (Efficacy Population) ............................................................................................ 189

Table A.4-2 Study 3002: QOL Questionnaire for Children and Teenager (Ages 8-12) (Safety Population)......................................................................................................... 199

Table A.4-3 Study 3002: QOL Questionnaire for Children and Teenager (Ages 4-7) (Safety Population) ..................................................................................................................... 235

Table A.4-4 Study 3002: Treatment-Emergent Adverse Events by System Organ Class, Preferred Term, and Maximum Relationship (Safety Population) ................................. 247

A.5 Study 3003 ..................................................................................................................... 270

Table A.5-1 Study 3003: Summary of Investigator’s (Surgeon’s) Clinical Assessment of Hemostatic Efficacy for Surgical Prophylaxis (Surgical Population) ............................ 270

Table A.5-2 Study 3003: FIX Activity Concentrations from Central Laboratory (All Subjects) 272

Table A.5-3 Study 3003: FIX Activity Concentrations from Local Laboratory (All Subjects) 287

Table A.5-4 Study 3003: Listing of Predicted and Intraoperative Estimated Blood Loss (Surgical Population) ..................................................................................................... 298

Table A.5-5 Study 3003: Listing of Predicted and Actual Transfusion Requirement (Surgical Population) ..................................................................................................... 300

- 140 -